Participation in the satellite symposia is free of charge. We thank the companies for their programme contributions and support!
Tuesday, 30 August 2022
Creating waves with PALYNZIQ® (pegvaliase): Three years of experience in Europe
12:45 – 13:45 CET, Hall Freiburg
Optimization of long-term management of patients with organic acidaemia (propionic and methylmalonic acidurias)
12:45 – 13:45 CET, Hall Jerusalem
Clinician and Patient Perspectives in the Management of Long-Chain Fatty Acid Oxidation Disorders
12:45 – 13:45 CET, Hall Porto
Wednesday, 31 August 2022
The FACEs* of Fabry disease
07:30 – 08:30 CET, Hall Jerusalem
Olipudase alfa: a new treatment for patients with ASMD
07:30 – 08:30 CET, Hall Porto
Classical Homocystinuria and Development of a New Enzyme Replacement Therapy
07:30 – 08:30 CET, Hall Kyoto
Reaching new hights in ex vivo gene therapy – therapeutic advances for early onset MLD patients
18.45-19.45 CET, Hall Jerusalem
Immunogenicity and Investigational AAV Gene Therapy
18.45-20.15 CET, Hall Porto
Thursday, 1 September 2022
Transformative science in action: Gene therapy for AADC deficiency
07.30-08.30 CET, Hall Jerusalem
Unmet Needs in Fabry disease: Where to go from here?
07.30-08.30 CET, Hall Porto
Pompe Disease: A Metabolic Disease Impacting Muscle and Potential Muscle-directed Gene Therapeutic Approaches
07.30-08.30 CET, Hall Kyoto
Improving the diagnostic pathway in MPS and treating Morquio A in the real world: best practice and findings from MARS
12.45-13.45 CET, Hall Freiburg
Pompe disease, a new era: the latest avalglucosidase alfa clinical data and early real-world experiences
12.45-13.45 CET, Hall Jerusalem
Optimizing early diagnosis in lysosomal storage diseases and innovation for the future
12.45-13.45 CET, Hall Porto
What matters most? Improving care in Lysosomal Storage Disorders (LSDs)
12.45-13.45 CET, Hall Kyoto
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